Development of a drug discovery platform for human islets

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DESCRIPTION (provided by applicant): Diabetes and diabetes-related co-morbidities are at epidemic proportions and an enormous burden to our healthcare system. Although there has been significant progress in defining the causative factors and molecular mechanisms involved in both type 1 and type 2 diabetes, it remains an overwhelming challenge to identify efficacious therapeutic modalities. Novel approaches and tools to accelerate research and development of additional therapeutics are urgently needed. Although the mechanisms are distinct, in both type 1 and type 2 diabetes there is a loss of pancreatic ßcells or ß-cell function, resulting in a complete or significant reduction in insulin production. Human islets are currently the most physiologically relevant system for the examination of potential therapeutics that modulate the insulin production/secretion, and factors that regulate growth and apoptosis of ß-cells. Biotech and pharmaceutical companies are in urgent need of novel contract research platforms and tools to accelerate their drug development programs that require human islets. The milestones of the Phase 1 application were successful and included the development of high throughput drug discovery platforms using "normal" and induced disease state models for screening purposes. In the current phase 2 application we will focus on the natural progression of these platforms which include the commercialization of novel 3D islet microtissues (pseudoislets) for research, establishing additional islet-based assays including, implementation of a quantitative high content imaging (QHCI) platform and finally validation of the current HT assays for contract research services using a small compound library screen. These products and services are novel and urgently needed by academic and pharmaceutical institutes; there is significant commercial potential.